Treatments

The following database contains products that have been granted orphan status, or have been market approval in the EU or the USA.

You can filter the content by clicking on the titles: Product, Indication, Sponsor and Date. Clicking once, will filter the column alphabetically from A – Z and a second click on the same title will filter from Z – A.

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Country Product Indication Sponsor Date
Recilisib sodium Acute Radiation Syndrome Acute radiation syndrome Onconova Therapeutics, Inc.;375 Pheasant Run;Newton, Pennsylvania, 18940 September 4, 2012
(E)-4-carboxystyryl-4-chlorobenzyl-sulfone, sodium salt Acute radiation syndrome Onconova Therapeutics, Inc.;375 Pheasant Run;Newton, PA, 18940 September 4, 2012
Caffeine and sodium benzoate Seizure prolongation in patients undergoing electroconvulsive therapy Luitpold Pharmaceuticals, Inc.;800 Adams Ave., Suite 100;Norristown, Pennsylvania, 19403 August 22, 2012
Triptorelin pamoate Precocious puberty Arbor Pharmaceuticals, LLC;6 Concourse Parkway, Suite 1800;Atlanta, Georgia, 30328 August 20, 2012
Diazepam (intranasal) Management of patients with acute repetitive seizures Acorda Therapeutics, Inc.;420 Saw Mill River Road;Ardsley, New York, 10502 July 31, 2012
Brilliant Blue G To selectively stain the thickened internal limiting membrane, which has formed onto the inner side of the retina in vitreo-retinal disorders Dutch Opthalmic Research Center;Scheijdelveweg 2, 3214 VN;Zuidland July 31, 2012
Dexamethasone sodium phosphate encapsulated in autologous erythrocytes Ataxia-Telangiectasia EryDel S.p.A.;Via Sasso 36, 61029 Urbino (PU); July 24, 2012
Acetyl-l-carnitine Fragile X syndrome Leadiant Biosciences, Inc.;9841 Washingtonian Blvd., Suite 500;Gaithersburg, Maryland, 20878 July 24, 2012
DNA plasmid vector expressing eIF5Ak50R protein, siRNA directed against the native eIF5A mRNA, and PEI (polyethyleneimine) Mantle cell lymphoma Senesco Technologies, Inc.;721 Route 202/206, Suite 130;Bridgewater, New Jersey, 08807 July 24, 2012
Autologous umbilical cord blood Pediatric (0-16 yrs old inclusive) cerebral palsy due to acquired brain injury Duke University;2301 Erwin Road;Durham, North Carolina, 27710 July 24, 2012
Recombinant Fusion Protein Myelodysplastic Syndrome Apogenix GmbH;Im Neuenheimer Feld 584, 69120;Heidelberg July 24, 2012
Antisense oligonucleotide targeted to human transthyretin (TTR) mRNA Hereditary ATTR amyloidosis Ionis Pharmaceuticals, Inc.;2855 Gazelle Court;Carlsbad, California, 92010 July 24, 2012
DNA plasmid vector expressing eIF5Ak50R protein, siRNA directed against the nastive eIF5A mRNA, and PEI (polyethyleneimine) Diffuse large B cell lymphoma Senesco Technologies, Inc.;721 Route 202/206, Suite 130;Bridgewater, New Jersey, 08807 July 24, 2012
Allogenic retinal epithelial cells transfected with plasmid vector expressing ciliary neurotrophic growth factor Macular telangiectasia type 2 (MacTel) Neurotech USA, Inc.;701 George Washington Highway;Lincoln, Rhode Island, 02865 July 24, 2012
Cantharidin Elastosis perforans serpiginosa Orenova Group, LLC;10 New King Street, Suite 106;White Plains, New York, 10604 July 24, 2012
Everolimus Hepatocellular Carcinoma Novartis Pharmaceuticals Corporation;One Health Plaza;East Hanover, New Jersey, 07936 July 23, 2012
N-acetylcysteine Prevention of ototoxicity caused by platinum-based chemotherapeutic agents used to treat pediatric cancers Edward A. Neuwelt, MD;Oregon Health & Science University, 3181 SW Sam Jackson Park Road - Mail Code L603;Portland, Oregon, 97239 July 19, 2012
N-(4-Fluoro-benzoyl)-L-arginyl-L-arginyl-[L-3-(naphthyl-alanyl]-L-cysteinyl-L-tyrosyl-L-citrullinyl-L-lysyl-D-lysyl-L-prolyl-L-trosyl-L-arginyl-L-citrullinyl-L-cysteinyl-L-aginine amide, cyclic (4-13)-disulfide For use to mobilize hematopoietic stem cells (HSCs) from the marrow to peripheral blood for collection for autologous or allogeneic transplantation BioLineRx, Ltd.;Modi'in Technology Park, 2 HaMa'ayan Street;Modi'in July 6, 2012
N-(4-Fluoro-benzoyl)-L-arginyl-L-arginyl-[L-3-(naphthyl-alanyl]-L-cysteinyl-L-tyrosyl-L-citrullinyl-L-lysyl-D-lysyl-L-prolyl-L-trosyl-L-arginyl-L-citrullinyl-L-cysteinyl-L-aginine amide, cyclic (4-13)-disulfide For use in combination with G-CSF to mobilize HSCs from the marrow to peripheral blood for collection for autologous or allogeneic transplantation BioLineRx, Ltd.;19 Hartum Street, P. O. Box 45158;Jerusalem, 91450 July 6, 2012
Benzoate Pediatric schizophrenia SyneuRx International (Taiwan) Corp.;1380 S. Marengo Ave;Pasadena, California, 91106 July 6, 2012
Adult adherent bone marrow-derived multipotent stem cells Hurler syndrome Athersys, Inc.;3201 Carnegie Avenue;Cleveland, Ohio, 44115 July 6, 2012
L-asparaginase encapsulated in red blood cells Pancreatic cancer ERYTECH Pharma;Batiment Adenine, 60 Avenue Rockefeller;lyon July 6, 2012
Antimesothelin-ADC (antibody drug conjugate) Malignant pleural mesothelioma Bayer HealthCare Pharmaceuticals, Inc.;340 Changebridge Road;Pine Brook, New Jersey, 07058 July 5, 2012
PG2 (Astragalus polysaccharides extracted and purified from the dry root of Astragalus membranaceus) Idiopathic thrombocytopenic purpura PhytoHealth Corporation;Fl. 5 No. 167, Taipei City, 105;Taipei ROC July 5, 2012
Bimagrumab Inclusion Body Myositis Novartis Pharmaceuticals Corp.;One Health Plaza;East Hanover, New Jersey, 07936 June 18, 2012